Tamibarotene Fails To Achieve Primary Endpoint In Syros’ MDS Trial

Syros Pharmaceuticals, a biopharma company focused on innovating frontline treatments for blood cancers, announced that its Phase 3 SELECT-MDS-1 trial did not achieve its primary goal. The study tested the efficacy of tamibarotene, Syros' exclusive oral RARα agonist, combined with azacitidine, in treating newly diagnosed, high-risk myelodysplastic syndrome (HR-MDS) patients who overexpress the RARA gene.

The complete response (CR) rate for the tamibarotene and azacitidine combination, based on an intent-to-treat analysis of the first 190 participants, was 23.8%, which did not show a significant difference compared to the 18.8% CR rate in the placebo group, with a p-value of 0.2084. 

Regarding safety, tamibarotene, when used alongside azacitidine in a group of 245 patients, was generally well-tolerated and showed a similar side-effect profile to that observed in previous studies. Syros also confirmed that the trial's outcome triggered a default event under its secured loan agreement with Oxford Finance LLC, as previously disclosed in its SEC filings.

“We are deeply disappointed by this outcome, particularly for the HR-MDS patients seeking a new treatment option for this challenging disease. We plan to stop the study, review the clinical data more thoroughly, and evaluate the next steps. We want to express our sincere appreciation for the patients, caregivers and healthcare professionals who took part in the SELECT-MDS-1 trial and to all the employees of Syros for their exceptional work on the tamibarotene program,” said Conley Chee, Chief Executive Officer of Syros.